SPINRAZA®

Generation 2+ antisense drug

SPINRAZA® (nusinersen) is the first and only approved treatment for all forms of spinal muscular atrophy (SMA) and is approved in more than 50 countries. A new clinical study, DEVOTE, has recently been initiated to assess if higher doses of SPINRAZA can provide even greater efficacy. For more information on the study (NCT04089566), please click here.

Due to a loss of, or defect in the survival motor neuron 1 (SMN1) gene, people with SMA do not produce enough SMN protein, which is critical for the maintenance of motor neurons. SPINRAZA is designed to selectively bind to and alter the splicing of the RNA from the paralog SMN2 gene, to produce full-length, fully functional SMN protein.

Spinal Muscular Atrophy is characterized by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness. Ultimately, individuals with the most severe type of SMA can become paralyzed and have difficulty performing the basic functions of life, like breathing and swallowing. People with Type 1 SMA, the most severe life-threatening form, produce very little SMN protein and do not achieve the ability to sit without support or live beyond two years without respiratory support. People with Type 2 and Type 3 produce greater amounts of SMN protein and have less severe, but still life-altering forms of SMA.